1 Introduction

1.1 Introduction to interim process statement

This interim process statement has been produced to guide the development of 'Evidence summaries: unlicensed and off-label medicines' (ESUOMs). It provides an overview of the key process principles and describes all stages of development for ESUOMs. These procedures are designed to ensure that a robust, quality-assured, commentary is developed for the NHS in an open, transparent and timely way, with appropriate input from key groups.

ESUOMs support the use of unlicensed and off-label medicines for individual patients, where there are good clinical reasons for the use of an off-label or unlicensed medicine, usually when there is no licensed medicine for the condition requiring treatment, or it is not appropriate for that individual. They should not be considered to promote the use of unlicensed medicines solely for economic reasons.

This interim process statement of ESUOMs will be superseded when the final process guide is issued later in 2012, following engagement and discussions with key groups.

1.2 Background to 'Evidence summaries: unlicensed and off-label medicines'

The National Institute for Health and Clinical Excellence (NICE) is part of the NHS. NICE's evidence-based guidance and other products help resolve uncertainty about which medicines, treatments, procedures, technologies and devices represent the best quality care and offer the best value-for-money for the NHS. Further information about NICE and its work is available on the NICE website (www.nice.org.uk).

The NICE Medicines and Prescribing Centre provides advice and support for delivering safety, efficiency and effectiveness in the use of medicines. The Medicines and Prescribing Centre is responsible for developing the ESUOMs.

ESUOMs will provide a summary of the published evidence for selected unlicensed or off-label medicines that are considered to be of significance to the NHS, where there are no clinically appropriate licensed alternatives. This will provide information for clinicians and patients to inform their decision-making and support the construction and updating of local formularies. The strengths and weaknesses of the relevant evidence will be critically reviewed within each ESUOM. Importantly, an ESUOM does not constitute NICE guidance and this will be clearly stated on each publication. This information from NICE will help to avoid potential duplication of effort where, currently, different NHS organisations produce similar products for their own local use.

The topics for ESUOMs are selected in collaboration with the existing NICE topic selection programme. Two types of topics are considered:

  • an unlicensed medicine: that is, a medicine that does not have a UK marketing authorisation and is not expected to do so in the next 2 years.

  • an off-label medicine: that is, a medicine with an existing UK marketing authorisation that is used outside the terms of its marketing authorisation, for example, by indication, dose, route or patient population.